Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

Completed

• Conditions: Sickle Cell Disease; Paroxysmal Nocturnal Hemoglobinuria; Rheologic Disease
• Interventions: Other: Imaging

• Registration Number: NCT01294891
• Lead Sponsor: Oregon Health and Science University

Brief Summary

The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. To test this question, the investigators will perform imaging tests (contrast ultrasound perfusion imaging) to look at the flow and function of these microvessels and compare this information to heart and kidney function. To further look at this question, patients who have PNH will be studied before and after starting a new drug (Soliris) that decreases damage to blood cells. In patients with sickle cell disease, patients will be studied at baseline (not during a pain crisis) and also during a pain crisis if one develops.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-02
• Study First Submitted: 2011-02-11
• Study First Submitted QC: 2011-02-11
• Study First Posted: 2011-02-14
• Status Verified: 2014-10
• Primary Completion: 2014-10
• Study Completion: 2014-10
• Last Update Submitted: 2014-10-27
• Last Update Posted: 2014-10-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

• Established diagnosis of PNH or SCD Age > 18 years old

Exclusion Criteria

• Pregnant or lactating women Presence of significant right to left shunting Allergy to ultrasound contrast agent Reactive airways disease Significant peripheral or coronary artery disease

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Sickle Cell Patients	Imaging	Patients with established SCD
Control	Imaging	Age matched healthy subjects
Paroxysmal Nocturnal Hemoglobinuria patients	Imaging	Patients with PNH

Primary Outcome Measures

Name	Time	Method
Microvascular perfusion	1 hour	1. Myocardial microvascular blood flow (MBF) and capillary blood velocity (CBV) on study day.; 2. Renal MBF on study day; 3. Skeletal muscle MBF and CBV on study day; 4. Myocardial MBF and CBV during hyperemia; 5. Skeletal muscle CBV during hyperemia

Trial Locations

Locations (1)

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States